We've heard for years that breakthroughs in stem cell research will revolutionize the way doctors treat chronic illnesses. But what will it take to make these promises a reality, and how close are we to new stem cell treatments?
In an exclusive interview with Healthline, Dr. Silviu Itescu, CEO of Mesoblast, explained how his regenerative medicine company is working to bring relief to those suffering from congestive heart failure, Crohn’s disease, rheumatoid arthritis, and other conditions using stem cells.
Itescu became the head of transplantation immunology at Columbia University in 1996, and is still on the cutting-edge of research in his field. By 2001, his team had shown in a preclinical study using rats that certain adult stem cells from bone marrow could be used to repair the heart. In 2013, Itescu received the inaugural Key Innovator Award from the Vatican’s Pontifical Council for Culture for his work on adult stem cell therapy.
Mesoblast is using mesenchymal precursor cells, or MPCs, to repair damaged hearts, control diabetes, and offer relief to those suffering from other inflammatory diseases.
The cells are extracted from the bone marrow of young, healthy donors. In a lab, magnets separate these one-in-100,000 cells from the others. Next, they are purified and grown in a medium. Within two months, a few thousand cells taken from a single donor multiply into the billions.
The cells have the ability to recognize injury and inflammation by honing in on secretions from damaged cells. The stem cells so far do not seem to provoke an immune response, and so they can be used by unrelated recipients, giving them a unique, off-the-shelf quality.
Treating Crohn’s, Diabetes, Rheumatoid Arthritis
By the end of this year, Mesoblast expects to have Phase 3 trials in all four of its key therapeutic areas: heart and vascular diseases, immunologic and inflammatory diseases, orthopedic diseases of the spine, and graft-versus-host-disease (GVHD).
The most promising of Mesoblast’s research focuses on therapies for those with congestive heart failure. Mesoblast’s partner for development and commercialization, Teva Pharmaceuticals, is recruiting 1,700 patients across multiple sites in the U.S. who suffer from class II and III heart failure.
Results of a 60-patient Phase 2 trial showed that of 15 heart failure patients treated with the highest dose of MPCs (150 million) with a single injection into the heart, no hospitalization was required for heart failure and no cardiac-related deaths occurred during three years of follow up. A similar trial is planned for even sicker patients with class IV heart failure who are currently on a left ventricular assisted device.
In essence, these are “the sickest of the sick,” said Itescu. Some may be waiting for heart transplants, but donor hearts are rare. Some may be so sick that a transplant is not possible.
Large-scale, Phase 3 studies cost a lot, Itescu said, upwards of $100 million to $200 million.
Itescu said that no company can go it alone. His publicly held company has forged partnerships not only with Teva but also with JCR Pharmaceuticals, a Japanese company, and Lonza, a global leader in biologics. Itescu even met last week with the Japanese prime minister.
Mesoblast will also be receiving incentives from the Singapore Economic Development Board.
“The only way to get stem cell technology into the market is a rigorous approach that costs a lot of money,” Itescu said.
FDA’s Blessing Badly Needed
FDA approval for stem-cell therapy is what is most urgently needed to move the field ahead, said Dr. Jeremy Rich, department chair of Stem Cell Biology and Regenerative Medicine at the Cleveland Clinic.
Cell-based therapy is still in its infancy, he said, and the FDA has not made many decisions about how to regulate it. That makes the process difficult for cautious pharmaceutical companies.
“What we lack right now is that success at a point where the FDA says, ‘Here you are, this is going to get approved,’” Rich told Healthline. “We need more experience about how to deliver (stem cells), and how to get them to the right place. Are we going to need some highly trained individuals who can do heart injections properly? Or can any cardiologist off the street do the same level of quality care. It’s as of yet unresolved.”
He said Mesoblast is doing the right thing by using its intellectual property to address an urgent need, for which standard drugs do not have much of an impact.
Indeed, while “cells are complicated machines,” they may be the only solution for the seriously sick, Rich said.
“It’s a paradigm shift that the pharmaceutical company has got to accept,” Itescu said.
Rich said other companies are attempting to do the same work as Mesoblast, and the FDA has been very proactive in working with them. “They are on a learning curve just like we are," he said. "It’s not so much a confrontational issue, it’s an issue of no one has the level of expertise to know what is safe and effective on these issues.”
He said a publicly held Cleveland-based company called Athersys is working in conjunction with Cleveland Clinic and Case Western Reserve University to develop a stem cell treatment for people with multiple sclerosis (MS).
Rich speculated that, eventually, large pharmaceutical companies will acquire companies that successfully bring stem cell treatments to market because it will be less expensive for them than building their own infrastructure.
“It’s like taking a giant cruise liner and telling them we have to totally change their direction and go in a different way. They don’t have the maps to go through those rock shoals," he said. "What they know how to do they do really well. They’re reluctant to get into it too far.”